Mitapivat

CAMBRIDGE, Mass. , Nov. 03, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that new data on mitapivat, an oral pyruvate kinase (PK) activator, will be featured in oral and poster presentations during the 67th American Society of Hematology (ASH) Annual Meeting and Exposition (ASH 2025) in Orlando, Florida , December 6-9, 2025 . “This year’s presentations at ASH highlight the growing momentum of our PK activation franchise, featuring new clinical and preclinical data that reinforce the therapeutic potential of mitapivat for patients with thalassemia, sickle cell disease, and PK deficiency – debilitating and life-threatening rare blood disorders with few or no treatment options,” said Sarah Gheuens , M.D., Ph.D., Chief Medical Officer and Head of R&D, Agios. “Building on these findings, we are also sharing research led by two of our advisory councils, each comprised of patients, caregivers, advocates, and physicians, that deepens our understanding of these rare diseases and helps guide the development of critical studies and resources tailored to patient needs. ASH provides a vital platform to showcase this important body of data and strengthen our connections with the hematology and rare disease communities.” Select presentations and publications at ASH 2025 will include: Two poster presentations on results from the ENERGIZE-T Phase 3 trial of mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia. The first is a subgroup analysis of patients with alpha-thalassemia, showing that 77.8% (7 of 9) of individuals in the mitapivat arm achieved the primary endpoint of transfusion reduction response, compared to 0% (0 of 3) in the placebo arm. Additionally, reductions in transfusion burden were observed in the mitapivat arm versus none in the placebo arm for all key secondary endpoints. The second highlights long-term results from the 17 patients who achieved transfusion independence with mitapivat during the double-blind phase of ENERGIZE-T, showing that the mean duration of their longest transfusion-free period was 30.5 weeks, with a maximum of 84.3 weeks, across the double-blind and ongoing open-label extension periods. An oral presentation with preclinical data from an investigator-led study demonstrating that mitapivat protects against cardiomyopathy (heart muscle disease) in a mouse model of beta-thalassemia, with this mechanism potentially linked to its activation of the PKM2 isoform (or variant) of the PK enzyme in the heart. Cardiomyopathy is a leading cause of morbidity and mortality in patients with hemolytic anemias. A poster presentation with positive findings from the ACTIVATE-Kids Phase 3 trial of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused. The trial met its primary endpoint, with the mitapivat arm showing a higher hemoglobin response rate compared to the placebo arm. Additionally, the mitapivat arm showed improvements in changes from baseline for hemoglobin concentration and markers of hemolysis (indirect bilirubin and lactate dehydrogenase) compared to the placebo arm. The safety results were consistent with the safety profile for mitapivat previously observed for adult patients with PK deficiency who are not regularly transfused. Research from two Agios-supported advisory councils, each comprised of patients, caregivers, advocates, and physicians, that builds on clinical and preclinical findings to help advance the scientific understanding of rare blood disorders. The first is a poster from the Thalassemia Advocacy Advisory Council , which showcases a global patient survey that identified key knowledge gaps about thalassemia, including awareness of complication risks at certain hemoglobin levels and the importance of regular monitoring in non-transfusion-dependent patients. The second is a publication-only study from the Red Cell Revolution, which highlights interim results of a qualitative survey assessing the physical, mental, and emotional impact of fatigue across patients with thalassemia, sickle cell disease, and PK deficiency, with cognitive impairment reported as the most bothersome manifestation of fatigue. The first is a subgroup analysis of patients with alpha-thalassemia, showing that 77.8% (7 of 9) of individuals in the mitapivat arm achieved the primary endpoint of transfusion reduction response, compared to 0% (0 of 3) in the placebo arm. Additionally, reductions in transfusion burden were observed in the mitapivat arm versus none in the placebo arm for all key secondary endpoints. The second highlights long-term results from the 17 patients who achieved transfusion independence with mitapivat during the double-blind phase of ENERGIZE-T, showing that the mean duration of their longest transfusion-free period was 30.5 weeks, with a maximum of 84.3 weeks, across the double-blind and ongoing open-label extension periods. The first is a poster from the Thalassemia Advocacy Advisory Council , which showcases a global patient survey that identified key knowledge gaps about thalassemia, including awareness of complication risks at certain hemoglobin levels and the importance of regular monitoring in non-transfusion-dependent patients. The second is a publication-only study from the Red Cell Revolution, which highlights interim results of a qualitative survey assessing the physical, mental, and emotional impact of fatigue across patients with thalassemia, sickle cell disease, and PK deficiency, with cognitive impairment reported as the most bothersome manifestation of fatigue. In total, 10 presentations and publications led by Agios and external collaborators will be shared at ASH 2025. ASH 2025 Accepted Abstracts Please refer to the ASH 2025 website for full session details and data presentation listings, and visit the Agios booth (#1661) onsite. About PYRUKYND® (mitapivat)U.S. INDICATIONPYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. U.S. IMPORTANT SAFETY INFORMATIONAcute Hemolysis: Acute hemolysis with subsequent anemia has been observed following abrupt interruption or discontinuation of PYRUKYND in a dose-ranging study. Avoid abruptly discontinuing PYRUKYND. Gradually taper the dose of PYRUKYND to discontinue treatment if possible. When discontinuing treatment, monitor patients for signs of acute hemolysis and anemia including jaundice, scleral icterus, dark urine, dizziness, confusion, fatigue, or shortness of breath. Hepatocellular Injury in Another Condition: In patients with another condition treated with PYRUKYND at a higher dose than that recommended for patients with PK deficiency, liver injury has been observed. These events were characterized by a time to onset within the first 6 months of treatment with peak elevations of alanine aminotransferase of >5× upper limit of normal (ULN) with or without jaundice. All patients discontinued treatment with PYRUKYND, and these events improved upon treatment discontinuation. Obtain liver tests prior to the initiation of PYRUKYND and monthly thereafter for the first 6 months and as clinically indicated. Interrupt PYRUKYND if clinically significant increases in liver tests are observed or alanine aminotransferase is >5x ULN. Discontinue PYRUKYND if hepatic injury due to PYRUKYND is suspected. Adverse Reactions: The most common adverse reactions including laboratory abnormalities (≥10%) in patients with PK deficiency were estrone decreased (males), increased urate, back pain, estradiol decreased (males), and arthralgia. Drug Interactions: Strong CYP3A Inhibitors and Inducers: Avoid concomitant use. Moderate CYP3A Inhibitors: Do not titrate PYRUKYND beyond 20 mg twice daily. Moderate CYP3A Inducers: Consider alternatives that are not moderate inducers. If there are no alternatives, adjust PYRUKYND dosage. Sensitive CYP3A, CYP2B6, CYP2C Substrates Including Hormonal Contraceptives: Avoid concomitant use with substrates that have narrow therapeutic index. UGT1A1 Substrates: Avoid concomitant use with substrates that have narrow therapeutic index. P-gp Substrates: Avoid concomitant use with substrates that have narrow therapeutic index. Hepatic Impairment: Avoid use of PYRUKYND in patients with moderate and severe hepatic impairment. Please see full Prescribing Information for PYRUKYND. About Agios: Fueled by Connections to Transform Rare Diseases™ At Agios, our vision is to redefine the future of rare disease treatment. Fueled by connections, we build trusted partnerships with communities – collaborating to develop and deliver innovative medicines that have the potential to transform lives. With a foundation in hematology, we combine biological expertise with real-world insights to advance a growing pipeline of rare disease medicines that reflect the priorities of the people we serve. Agios is a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts . To learn more, visit www.agios.com and follow us on LinkedIn and X. Cautionary Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of PYRUKYND® (mitapivat); Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®; Agios’ plans regarding future data presentations; and the potential benefits of Agios’ strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of pandemics or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of AG-236, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Contacts: Investor Contact Morgan Sanford , Vice President, Investor Relations Agios Pharmaceuticals morgan.sanford@agios.com Media Contact Eamonn Nolan , Senior Director, Corporate Communications Agios Pharmaceuticals eamonn.nolan@agios.com Source: Agios Pharmaceuticals, Inc.

$12.9 million in third quarter PYRUKYND® (mitapivat) net revenuesPDUFA goal date for PYRUKYND U.S. sNDA in thalassemia set for December 7, 2025CHMP adopted positive opinion for PYRUKYND in thalassemia; EC decision expected by early 2026RISE UP Phase 3 trial topline results in sickle cell disease by year-end; potential U.S. commercial launch in 2026Phase 2b tebapivat trial in lower-risk MDS fully enrolled; topline results expected in early 2026$1.3 billion dollars in cash, cash equivalents and marketable securities as of September 30, 2025 CAMBRIDGE, Mass., Oct. 30, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced financial results and updates for the third quarter ended September 30, 2025. “As we approach year-end, we remain focused on our two key PYRUKYND milestones – the potential U.S. approval in thalassemia and the topline results from the RISE UP Phase 3 trial in sickle cell disease. Our recent engagements with these communities have underscored the urgent need for innovation and PYRUKYND’s potential to address critical gaps in care for these serious and life-threatening diseases,” said Brian Goff, Chief Executive Officer, Agios. “We continued strong execution across our rare disease portfolio in the third quarter, including the completion of enrollment in our Phase 2b tebapivat trial for lower-risk MDS. We look forward to building on this strong momentum and remain steadfast in our commitment to delivering meaningful progress for the patients we serve.” Third Quarter 2025 and Recent Corporate HighlightsCommercial Performance – PYRUKYND® (mitapivat) Generated $12.9 million in net revenue for the third quarter of 2025, representing an increase of 44 percent from $9.0 million in the third quarter of 2024 and a 3 percent increase from $12.5 million in the second quarter of 2025. 262 unique patients completed prescription enrollment forms, representing an increase of 6 percent over the second quarter of 2025.149 patients are on therapy in the U.S., inclusive of new starts and continued therapy, representing an increase of 5 percent over the second quarter of 2025. R&D HighlightsPYRUKYND (mitapivat) Thalassemia – United States – U.S. Food and Drug Administration (FDA) extended the Prescription Drug User Fee Act (PDUFA) goal date for the supplemental New Drug Application (sNDA) of PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia by three months, to December 7, 2025. Extension was triggered by FDA request for a Risk Evaluation and Mitigation Strategy (REMS) to address the potential risk of hepatocellular injury described in the original application.Extension was not the result of new or additional efficacy or safety data requested by the FDA or submitted by Agios.U.S. commercial launch preparations remain underway, and the application remains under active FDA review. Europe – Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of PYRUKYND in adults for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia. A final decision from the European Commission is expected by early 2026. Gulf Cooperation Council (GCC) – PYRUKYND received approval in Saudi Arabia for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. Commercial launch activities are underway in Saudi Arabia in partnership with NewBridge Pharmaceuticals. In the United Arab Emirates, PYRUKYND thalassemia regulatory application remains under active review. Sickle Cell Disease – Topline results from the RISE UP Phase 3 trial of mitapivat in sickle cell disease are expected by year-end, potentially supporting a U.S. commercial launch in 2026. Tebapivat Lower-risk Myelodysplastic Syndromes (LR-MDS) – Completed patient enrollment in the Phase 2b trial of tebapivat in LR-MDS. Following findings from the Phase 2a trial, the Phase 2b trial is evaluating three higher daily doses (10 mg, 15 mg, and 20 mg) versus placebo over 24 weeks. Topline results from this trial are expected in early 2026. Third Quarter 2025 Financial Results For the quarter ended September 30, 2025, net loss was $103.4 million dollars, compared to net income of $947.9 million dollars for the quarter ended September 30, 2024. The net income in the third quarter of 2024 was due to the milestone payment from Servier and the sale of royalty rights to Royalty Pharma, both of which were recorded in that quarter. Net product revenue from sales of PYRUKYND for the third quarter of 2025 was $12.9 million, compared to $9.0 million for the third quarter of 2024.Cost of sales for the third quarter of 2025 was $1.7 million.Research and Development (R&D) Expenses were $86.8 million for the third quarter of 2025, an increase of $14.3 million compared to the third quarter of 2024. The year-over-year increase was primarily driven by increased clinical trial costs associated with the PK activation franchise. Selling, General and Administrative (SG&A) Expenses were $41.3 million for the third quarter of 2025, representing an increase of $2.7 million compared to the third quarter of 2024, primarily driven by disciplined investment in preparation for the potential U.S. commercial launch of PYRUKYND in thalassemia.Cash, cash equivalents and marketable securities as of September 30, 2025, were $1.3 billion compared to $1.5 billion as of December 31, 2024. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to prepare for potential PYRUKYND commercial launches in thalassemia and sickle cell disease, advance existing clinical programs, and opportunistically expand its pipeline through both internally and externally discovered assets. Conference Call Information Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s third quarter 2025 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company's website (www.agios.com) under the “Events & Presentations” tab. A replay of the webcast will be available on the company’s website approximately two hours after the event. About Agios: Fueled by Connections to Transform Rare Diseases™ At Agios, our vision is to redefine the future of rare disease treatment. Fueled by connections, we build trusted partnerships with communities – collaborating to develop and deliver innovative medicines that have the potential to transform lives. With a foundation in hematology, we combine biological expertise with real-world insights to advance a growing pipeline of rare disease medicines that reflect the priorities of the people we serve. Agios is a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts. To learn more, visit www.agios.com and follow us on LinkedIn and X. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of PYRUKYND® (mitapivat), tebapivat, AG-236 and AG-181; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, tebapivat, AG-236 and AG-181; Agios’ expectations for the review of marketing applications for PYRUKYND by regulatory agencies, including the FDA and European Commission; Agios’ strategic vision and goals, including its key milestones for 2025; and the potential benefits of Agios’ strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of pandemics or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of AG-236, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Consolidated Balance Sheet Data (in thousands) (Unaudited) September 30, 2025 December 31, 2024 Cash, cash equivalents, and marketable securities$1,257,201 $1,532,031 Accounts receivable, net 5,029 4,109 Inventory 32,034 27,616 Total assets 1,385,705 1,663,199 Stockholders' equity 1,284,330 1,540,956 Consolidated Statements of Operations Data(in thousands, except share and per share data)(Unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 2025 2024 Revenues: Product revenue, net$12,880 $8,964 $34,061 $25,768 Total revenue 12,880 8,964 34,061 25,768 Operating expenses: Cost of sales$1,679 $783 $4,466 $2,905 Research and development 86,796 72,455 251,479 218,476 Selling, general and administrative 41,274 38,537 128,670 105,087 Total operating expenses 129,749 111,775 384,615 326,468 Loss from operations (116,869) (102,811) (350,554) (300,700)Gain on sale of contingent payments — 889,136 — 889,136 Milestone payment from gain on sale of oncology business — 200,000 — 200,000 Interest income, net 13,369 13,059 43,969 30,068 Other income, net 67 1,651 1,843 4,864 Net (loss) income before taxes (103,433) 1,001,035 (304,742) 823,368 Income tax expense — 53,120 — 53,120 Net (loss) income$(103,433) $947,915 $(304,742) $770,248 Net (loss) income per share - basic$(1.78) $16.65 $(5.27) $13.58 Net (loss) income per share - diluted$(1.78) $16.22 $(5.27) $13.38 Weighted-average number of common shares used in computing net (loss) income per share – basic 58,139,277 56,939,403 57,846,173 56,709,318 Weighted-average number of common shares used in computing net (loss) income per share – diluted 58,139,277 58,432,796 57,846,173 57,581,382 Contacts: Investor ContactMorgan Sanford, Vice President, Investor Relations Agios Pharmaceuticalsmorgan.sanford@agios.com Media ContactEamonn Nolan, Senior Director, Corporate CommunicationsAgios Pharmaceuticalseamonn.nolan@agios.com